EU rules "mean children can't get life-saving cancer drugs"

LONDON (Reuters) - Children with cancer are being denied
access to potentially life-saving medicines because European
Union rules allow drug firms to waive the need to test some
drugs in pediatric trials, researchers said on Monday.

Calling for a loophole in EU legislation to be closed,
scientists and specialists in childhood oncology led by
Britain's Institute for Cancer Research (ICR) said changing the
rules could extend or save many young lives.

"Many cancer drugs developed for adults could be effective
in children if we were able to test them in clinical trials,"
Dr. Louis Chesler, an ICR researcher and a pediatric oncologist
at London's Royal Marsden hospital, told reporters at a
briefing.

Currently, pharmaceutical companies developing new cancer
medicines can gain exemptions from carrying out expensive
testing of those drugs in patients under age 18, even where a
drug's mechanism of action suggests it could work in children.

As a result, campaigners and scientists say, there are often
long delays before new drugs are available for children, and
some are never licensed for pediatric use at all.

"Kids get hand-me-downs in many areas of life," said
Chesler. "But cancer drugs should not be an area where kids get
hand-me-downs."

Experts say the system is out of date because it is based on
previous thinking about cancer as a disease specific to the
organ - such as lung, breast or bowel cancer - rather than on
latest scientific knowledge showing cancers are driven by
genetic changes that can happen in many parts of the body and in
various types and ages of people.

Drugs have been approved for treating adult lung and other
cancers driven by mutations in ALK or EGFR genes, for example,
but the companies have been granted waivers from testing them in
children - even though ALK and EGFR mutations have been shown to
play a role in some childhood cancers.

"The current system is failing to provide children with
access to new treatments that could add years to their lives,"
Alan Ashworth, ICR's chief executive, told the briefing.

"We're not blaming the EU legislation for this - but it
needs rapidly on be modernized," he added.

Research carried out by the ICR together with the European
Consortium for Innovative Therapies for Children with Cancer
(ITCC), based in France, found that of 28 cancer drugs approved
for adult marketing authorization in Europe since 2007, 26 work
in a way that is relevant for childhood cancers.

Yet 14 have been exempted from being tested in children
because the specific adult condition for which the drug is
developed does not occur in children.

The ICR and other international cancer researchers and
campaigners want the EU to abolish drugmakers' right to an
opt-out - a change that would compel them to test a new drug's
potential to treat children's cancers as well as adults'.

Ashworth also said he'd like to see stronger financial
incentives to ensure a decent return for drugmakers who take on
the challenge of developing new drugs that are only likely to be
able to be used in a small number of patients.